In an R&D field typically characterized by slow progress and incremental gains, Isis Pharmaceuticals ($ISIS) jumped out of the ADA pack in Chicago with some stellar mid-stage data from a very small study of a gene-silencing treatment that slashed triglycerides and pumped up insulin sensitivity in diabetics. And now the biotech is hustling ahead into a Phase III pivotal trial to see if it can match those numbers in a much bigger group of patients.

After 13 weeks of weekly injections of ISIS-APOCIIIRx--designed to hit the 'off' switch on a gene that produces the apolipoprotein C-III protein, involved in triglyceride regulation--there was a 72% plunge in fatty particles and a 40% spike in HDL, or good cholesterol, along with improved insulin sensitivity among the 11 patients with Type 2 diabetes in the study.

Investors bid up shares in the biotech by 27% yesterday, impressed by the potential of a drug that could prove significantly better than Kynamro, a drug that Isis and Sanofi ($SNY) market in the U.S. European regulators, though, turned the drug down after fretting over liver toxicity. And Isis carefully noted that there were no signs of an elevation in liver enzymes, a common red flag on toxicity, or any other adverse events worth worrying about.

TheStreet's Adam Feuerstein also notes that there are 50,000 patients with extremely high levels of triglycerides, many of whom could be treated now with Amarin's Vascepa or niacin or fibrates. And Isis is designing its pivotal study to see if they could benefit from this experimental therapy.

Isis produced data that were "well beyond expectations and suggest an unprecedented treatment benefit in Type 2 diabetics with (high cholesterol levels)," BMO Capital Markets analyst Jim Birchenough noted, according to a report in Reuters. "A blockbuster opportunity could emerge," he added, guessing that this new therapy could garner roughly $850 million a year.

"Our focus is to bring ISIS-APOCIII to the market for patients with severe hypertriglyceridemia. These are patients who cannot reduce their triglycerides to safe levels with currently available medicines. We plan to report data from our ongoing Phase 2 program in very high triglyceride patients later this summer evaluating ISIS-APOCIII in combination with fibrates and as a monotherapy," said Richard Geary, senior VP of clinical development at Isis. "In the study we are reporting today, we observed rapid and prolonged reductions of apoC-III, triglycerides and other lipid parameters, as well as improvements in glucose control and insulin sensitivity. These data suggest that ISIS-APOCIII could provide therapeutic benefit to patients with high triglycerides who are insulin insensitive, including patients who are obese or have Type 2 diabetes. In addition, the positive effect of ISIS-APOCIII on all atherogenic lipid parameters measured and the observed increase in HDL, significantly enhances the potential profile of the drug."

- here's the press release
- read the story from TheStreet
- and the report from Reuters